A Research Study of How Well Macimorelin Works to Find Out if Children Have a Lack of Growth Hormone and How Safe it is (DETECT)
Growth Hormone Deficiency
About this trial
This is an interventional diagnostic trial for Growth Hormone Deficiency focused on measuring childhood-onset growth hormone deficiency, diagnosis of growth hormone deficiency, diagnosis of childhood-onset growth hormone deficiency, diagnostic test for growth hormone deficiency
Eligibility Criteria
Inclusion Criteria:
- Informed consent of subject, parent(s) or legally acceptable representative (LAR) of subject and child assent, if appropriate, must be obtained before any trial-related activities. Trial-related activities are any procedures that are carried out as part of the trial, including activities to determine suitability for the trial.
- Male and female pediatric subjects from 2 to less than 18 years of age at the time of signing informed consent.
- Indication for the performance of growth hormone stimulation test.
- Presence of a height measurement minimum 6 and maximum 18 months prior to screening.
Exclusion Criteria:
- Established diagnosis of a disease that is sufficient to explain growth deficiency or metabolic disorders that are also associated with short stature (e.g., Turner syndrome, skeletal dysplasia's, celiac disease, etc.).
- Ongoing growth hormone therapy.
- Presence of hypothyroidism and/or adrenal insufficiency without adequate and stable replacement therapy treatment for at least 30 days prior to first GHST.
- Treatment with drugs directly affecting the pituitary secretion of somatotropin (e.g., somatostatin analogues, clonidine, levodopa and dopamine agonists) or provoking the release of somatostatin (antimuscarinic agents e.g., atropine).
- Medical history of ongoing clinically symptomatic psychiatric disorders.
- 2nd or 3rd degree atrioventricular-block, prolongation of the QRS complex over 120 milliseconds, prolongation of the QTc interval over 450 milliseconds, or any other clinically significant abnormal electrocardiogram results at the V2 pre-dose electrocardiogram (ECG) as judged by the investigator.
- Previous participation in this trial. Participation is defined as signed informed consent.
- Participation in any clinical trial of an approved or non-approved investigational medicinal product within 30 days before screening.
- Known or suspected hypersensitivity to trial product(s) or related products;
- Any disorder, which in the investigator's opinion might jeopardize subject's safety or compliance with the protocol.
- Concomitant treatment with any drugs that might prolong QT/QTc Note: A subject who receives such treatment will not be a candidate for this study, if his/her condition does not allow for a treatment-free period of at least 5 elimination half-lives of the drug that might prolong QT/QTc before the GHST;
- Elevation of laboratory parameters indicating hepatic or renal dysfunction or damage (aspartate amino transferase (AST), alkaline phosphatase (ALT), gamma-glutamyl transferase (GGT) > 2.5 x upper limit of normal (ULN); creatinine or bilirubin > 1.5x ULN);
- Current active malignancy other than non-melanoma skin cancer;
- Female of child-bearing potential and not using an adequate contraceptive method (adequate contraceptive measures as required by local regulation or practice).
- Male of reproductive age who or whose partner(s) is not using an adequate contraceptive method (adequate contraceptive measures as required by local regulation or practice).
- Lack of ability or willingness to give informed consent by the subject and/or his/her legal representative;
- Anticipated non-availability for trial visits/procedures.
Sites / Locations
- Angel Wing Clinic For Children With DiabetesRecruiting
- Pediatric Endocrine Associates, p.c.Recruiting
- John Hopkins All Children's HospitalRecruiting
- Emory Healthcare-Children's CenterRecruiting
- St. Luke's Children's EndocrinologyRecruiting
- University of Minnesota, Masonic Children's HospitalRecruiting
- The Children's Mercy Hospital - BroadwayRecruiting
- Icahn School of Medicine at Mount SinaiRecruiting
- UNC HospitalsRecruiting
- Diabetes and Glandular Disease Clinic, PA
- Multicare Health SystemRecruiting
- JSC Maritime HospitalRecruiting
- National Institute of EndocrinologyRecruiting
- TSMU Givi Jvania Pediatric Academic ClinikRecruiting
- Evangelisches Klinikum BethelRecruiting
- Ospedale Pediatrico G. SalesiRecruiting
- Azienda Ospedaliero-Universitaria Anna MeyerRecruiting
- Osp. dei Bambini V. Buzzi, ASST Fatebenefratelli SaccoRecruiting
- Azienda Ospedaliero-Universitaria di Parma Ospedale dei Bambini Pietro Barilla, Clinica PediatricaRecruiting
- IRCCS Ospedale Pediatrico Bambino GesùRecruiting
- MED-POLONIA Sp.z o.o.Recruiting
- Kliniczny Szpital Wojewodzki nr 2 im. Sw. Jadwigi Krolowej w RzeszowieRecruiting
- SPSK Nr 1 im. prof. Tadeusza Sokolowskiego PUM
- Uniwersytecki Szpital Kliniczny im. Jana Mikulicza Radeckiego we WroclawiuRecruiting
- Cen Med de Diagn si Trat Amb NEOMEDRecruiting
- Institutul de Endocrinologie "C.I. Parhon"Recruiting
- Sana MonitoringRecruiting
- Medicover HospitalsRecruiting
- Spitalul Clinic Judetean de Urgenta "Sf. Apostol Andrei" ConstantaRecruiting
- Spitalul Clinic Judetean de Urgenta "Sf. Spiridon" IasiRecruiting
- Spitalul Cl. de Urgenta pentru Copii Louis Turcanu TimisoaraRecruiting
- Spitalul Clinic Judetean MuresRecruiting
- University children's clinic Belgrade - Department of EndocrinologyRecruiting
- Clinical Center Nis - Clinic for Children's Internal MedicineRecruiting
- Institute for Child and Youth Health Care of Vojvodina - EndocrinologyRecruiting
- National Institute of Endocrinology and DiabetologyRecruiting
- Univerzitetni Klinicni Center Ljubljana - PediatricsRecruiting
Arms of the Study
Arm 1
Arm 2
Active Comparator
Active Comparator
standard GHST order randomized: arginine - clonidine
standard GHST order randomized: clonidine - arginine
At visit 2 (V2), all subjects will perform the macimorelin GHST and will be randomized 1:1 to the order of the clonidine and arginine GHSTs at visit 3 (V3) and visit 4 (V4). In this arm, those subjects will be presented which will have been randomized to the arginine GHST at V3 and the clonidine GHST at V4. At visit 5 (V5) all subjects will perform the macimorelin GHST.
At V2, all subjects will perform the macimorelin GHST and will be randomized 1:1 to the order of the clonidine and arginine GHSTs at V3 and V4. In this arm, those subjects will be presented which will have been randomized to the clonidine GHST at V3 and to the arginine GHST at V4. At V5 all subjects will perform the macimorelin GHST.